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Updated: Mar 19, 2026

Use of Ultra-high Field MRI in Small Rodent Models of Polycystic Kidney Disease for In Vivo Phenotyping and Drug Monitoring
Published on: June 23, 2015
[AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE: HOW AND WHY SHOULD WE IDENTIFY THE PATIENTS "RAPIDLY PROGRESSING" TO
Insights
Autosomal dominant polycystic kidney disease (ADPKD) is an inherited condition causing kidney cysts and often leading to kidney failure. Identifying rapid progressors aids in early intervention and management to slow disease advancement.
Area of Science:
- Nephrology
- Genetics
- Internal Medicine
Context:
- Autosomal dominant polycystic kidney disease (ADPKD) is a prevalent inherited disorder.
- ADPKD is characterized by bilateral kidney cyst development, frequently leading to end-stage renal disease (ESRD).
- Early identification of "rapid progressors" is crucial for managing ADPKD and mitigating ESRD risks.
Purpose:
- To outline current methods for assessing ADPKD progression.
- To discuss the efficacy of tolvaptan in slowing cyst growth and GFR decline.
- To review current ADPKD management strategies focused on risk factor modification.
Summary:
- ADPKD progression is evaluated using clinical scores (PROPKD-Score), biological markers (GFR decline, creatinine doubling), and radiological criteria (TKV, TKV growth rate).
- While no cure exists, vasopressin-2 receptor antagonists like tolvaptan show promise in slowing renal cyst growth and GFR decline.
- Current ADPKD management involves addressing risk factors such as hydration, blood pressure, and smoking cessation.
Impact:
- This information aids clinicians in identifying high-risk ADPKD patients for timely intervention.
- Understanding progression markers and treatment options can improve patient outcomes and slow the development of ESRD.
- The review highlights the importance of a multi-faceted approach to ADPKD management, combining risk factor control with emerging therapies.
Abstract:
Autosomal dominant polycystic kidney disease (ADPKD) is a common inherited disease characterised by the progressive development of multiple and bilateral cysts in kidneys and other organs. Most patients with ADPKD will develop, sooner or later, end-stage renal disease (ESRD). The morbidity and mortality associated with ESRD prompt physicians to identify early ADPKD patients considered as "rapid progressors", who have the greatest risk to rapidly develop ESRD. The rate of progression can be assessed by clinical--especially with the "predicting renal outcome in polycystic kidney disease score" (PROPKD-Score)-, biological (a decline of the glomerular filtration rate (GFR) of 4.4-5.9 ml/min/year and/or the doubling of serum creatinine within a 36-month period), or radiological criteria (total kidney volume (TKV) adjusted for the size > 600 cc/m and/or TKV annual growth rate > 5 %). Nowadays, there is no curative treatment for ADPKD. However, vasopressin-2 receptor antagonists, such as tolvaptan, appear to slow down the growth of renal cysts and the slope of GFR decline. The current management of ADPKD patients is mostly based on correcting the risk factors for progression, i.e. encouraging (over)-hydration, normalizing blood pressure, stimulating smoking cessation.
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